BPC February 11 update

​Infinity INFI releases updated bladder cancer data +13%; ​Amicus FOLD misses late-stage Pompe trial goal -19%

Price and Volume Movers

Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) shares closed up 13% to $4.47 following the presentation of data from its Phase 2 MARIO-trial evaluating eganelisib in combination with nivolumab (Opdivo) in platinum-refractory, I/O naïve patients with advanced urothelial cancer. In the overall population, the combination arm demonstrated an overall response rate (ORR) of 30% compared with 25% in the nivolumab monotherapy arm. When broken down into population groups, patients in the PD-L1 negative patient population saw the greatest benefit with an ORR of 26% for the combo compared with 14% for nivolumab monotherapy. The company also announced after hours that it has commenced an underwritten public offering of its common stock, which has seen shares trade up a further 5%.

Amicus Therapeutics (Nasdaq: FOLD) shares have fallen 19% after hours to $15.15 following the release of top-line data from its Phase 3 Propel trial of AT-GAA for the treatment of late-onset Pompe disease (LOPD). The primary endpoint of the change in 6-minute walk distance as compared with baseline measurements, was not met. While numerically greater, with patients walking on average 21 meters farther compared to 7 meters with those treated with current standard of care alglucosidase alfa, statistical significance for superiority was not achieved (p=0.072). The company still intends to complete its Biologics License Application (BLA) for approval with the FDA, which it commenced late-2020. Completion of the BLA submission is expected next quarter.

VYNE Therapeutics Inc. (NASDAQ:VYNE) announced a reverse stock split at a ratio of 1-for-4 will become effective on February 16, 2021.

Protalix BioTherapeutics, Inc. (NYSE:PLX) shares are trading down 9% after hours to $5.25 following news of a public offering of 6.5m shares of its common stock.

Diffusion Pharmaceuticals Inc. (NASDAQ:DFFN) announced that H.C. Wainwright & Co. will purchase 9,756,098 shares of common stock at a price of $1.025 per share, for gross proceeds of $10m. Shares are trading down 13% to $1.17 after hours.

Corvus Pharmaceuticals, Inc. (NASDAQ:CRVS) also announced that it has commenced an underwritten public offering of shares of its common stock. Shares are currently trading down 7% to $3.65 after hours.

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Major price movers (stocks priced > $1.00, volume > 50k listed on NASDAQ/NYSE):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

Ayala Pharmaceuticals, Inc. (AYLA): $19.95; +53%.

ESSA Pharma Inc. (EPIX): $28.65; +48%.

ADiTx Therapeutics, Inc. (ADTX): $5.26; +28%.

Monopar Therapeutics Inc. (MNPR): $10.60; +26%.

Tonix Pharmaceuticals Holding Corp. (TNXP): $2.00; +23%.

DECLINERS:

Anchiano Therapeutics Ltd. (ANCN): $4.81; -40%.

Zynerba Pharmaceuticals, Inc. (ZYNE): $6.11; -30%.

Pulmatrix, Inc. (PULM): $2.01; -24%.

Bio-Path Holdings, Inc. (BPTH): $9.50; -19%.

Intec Pharma Ltd (NTEC): $6.66; -15%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACER – Acer Therapeutics Inc.
ACER-001
Urea cycle disorder (UCD)

NDA Filing Pre-NDA meeting with FDA due 2Q 2021 with NDA filing due mid-2021.
$45.6 million

AGTC – Applied Genetic Technologies Corporation
AGTC-501
X-linked Retinitis Pigmentosa (XLRP)

Phase 1/2 Phase 2/3 (VISTA) six-month interim analysis 3Q 2022. Phase 1/2 interim analysis (SKYLINE) due 4Q 2021 with 12-month data due 3Q 2022.
$247.8 million

ALNY – Alnylam Pharmaceuticals Inc.
Lumasiran (ALN-GO1) ILLUMINATE-C
Impaired renal function

Phase 3 Phase 3 top-line data due mid-2021.
$17.8 billion

ALVR – AlloVir Inc.
ALVR105
Multi-virus prevention trial

Phase 2 Phase 2 initial data from open-label phase due 2H 2021.
$2.4 billion

AVXL – Anavex Life Sciences Corp.
ANAVEX 2-73 - AVATAR
Rett syndrome

Phase 2 Phase 2 top-line data due 1H 2021.
$967.3 million

CLSN – Celsion Corporation
GEN-1 OVATION 2
Ovarian cancer

Phase 2 Phase 2 overall response rate (ORR) data due 2021. PFS data 4Q 2022.
$156.7 million

EPIX – ESSA Pharma Inc.
EPI-7386
Castrate resistant prostate cancer

Phase 1 Phase 1 initial data presented at ASCO GU February 11, 2021.
$792.2 million

FOLD – Amicus Therapeutics Inc.
AT-GAA - Propel
Pompe Disease

BLA Filing Phase 3 top-line data did not meet primary endpoint - February 11, 2021. Rolling BLA filing to be completed 2Q 2021.
$2.9 billion

INFI – Infinity Pharmaceuticals Inc.
IPI-549 + Nivolumab (MARIO-275)
Urothelial cancer

Phase 2 Phase 2 data announced February 11, 2021. Overall response rate 30% in combo arm vs 25% in nivolumab monotherapy arm. In PD-L1 Negative sub group, overall response rate 26% in combo arm vs 14% in nivolumab monotherapy arm.
$272.2 million

IVA – Inventiva S.A.
Lanifibranor
Nonalcoholic fatty liver disease (NAFLD)

Phase 2 Phase 2 data to be published in 2021.
$592.6 million

OTIC – Otonomy Inc.
OTIVIDEX
Ménière's disease

Phase 3 Phase 3 trial did not achieve the primary endpoint.
$140.1 million

OTLK – Outlook Therapeutics Inc.
NORSE 3 ONS-5010 (open-label)
Wet age-related macular degeneration (wet AMD)

Phase 3 Phase 3 top-line data due 2Q 2021.
$368.4 million

RARE – Ultragenyx Pharmaceutical Inc.
GTX-102
Angelman Syndrome

Phase 1/2 Phase 1/2 further data due 2H 2021.
$9.4 billion

SGEN – Seagen Inc.
Enfortumab vedotin and Keytruda EV-103
Urothelial cancer

Phase 1/2 Phase 1/2 Cohort K enrollment to be completed by end of 2021.
$27.8 billion